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A contemporary review published in the American Journal of Hematology provides updated recommendations for allogeneic hematopoietic stem-cell transplantation.
For patients with myelodysplastic neoplasms and myelofibrosis, a standard treatment option is allogeneic hematopoietic stem-cell transplantation (HCT). Even with increased use and advancements in HCT, questions remain about transplant management, timing, and patient selection.
Researchers conducted a review and meta-analysis of the latest HCT research and recommendations for the American Journal of Hematology.
Patient Selection
When assessing if a patient is a good fit for HCT, clinicians should evaluate disease- and patient-related risk factors. One of the most important patient-related factors is age.
“Patients should be in good general health status and preferentially less than 70 years old,” corresponding author Nicolaus Kröger, MD, told Physician’s Weekly in an interview. “But [patients] older than 70 can also be accepted on an individual basis.”
The researchers also recommend conducting frailty assessments and looking for pre-existing conditions like iron overload, which may require chelation therapy.
Other disease-related factors that help determine candidacy for HCT include:
- Genomic profile, especially for somatic mutations
- Risk stratification
- Risk for certain complications
Dr. Kröger stated that these factors can be used to determine a predicted life expectancy, which can indicate whether HCT is a suitable treatment option.
“The important thing is to determine the risk for the disease. There are specific scoring systems like DIPSS or MIPSS70, which allow clinicians to predict life expectancies,” Dr. Kröger says. “If life expectancy is less than five years, allogeneic stem cell transplantation should be considered, and transplant-specific score should be determined (like Myelofibrosis Transplant Scoring System) to predict outcome after allogeneic stem cell transplantation.”
HCT Timing
The timing of HCT impacts treatment effectiveness; however, there are no general guidelines on timing, as it depends on a patient’s profile. The researchers wrote that recent advances “underscore the critical role of nuanced decision making using genomic insights together with disease-, patient-, and transplant-related factors, offering potential for precision medicine approaches and improved clinical outcomes.”
Citing a landmark study, the researchers suggested that delaying HCT maximizes survival for patients under 40 or with low or intermediate-1 IPSS. By contrast, immediate HCT may maximize survival for those with intermediate-2 or high International Prognostic Scoring System risk. Donor availability and patient preferences can also influence the HCT timeline.
Transplant Management
Selecting the right donor can impact effectiveness, with younger donors often yielding better results. The ideal option is matched-donor siblings, except for when age or comorbidities exclude these donors.
In addition to donor selection, post-transplant care matters, and there is an established protocol to maximize outcomes.
“The molecular follow-up is not extensive and should include only the underlying driver mutation, which is either JAK2, CALR, or MPL,” Dr. Kröger says. “Here, commercially available tests exist, and persistence of this maker in peripheral blood suggests residual disease. In such cases, the immunosuppressive therapy can be carefully reduced to enhance the graft versus myelofibrosis effect, resulting in an elimination of the residual disease detected by the molecular testing.”
As relapse is an issue, molecular monitoring is recommended one month after HCT and every three months for the first year.
“Optimizing conditioning regimens, particularly tailoring the intensity to individual patient profiles, requires further exploration through prospective trials,” the researchers noted. “The role of MRD in guiding preemptive intervention before and after HCT is a promising area that needs standardization and broader implementation.”
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